Expert queries high cost CF drug for few

Ivacaftor (Kalydeco, Vertex) was approved by the TGA last week for people with CF, aged six and older, who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The treatment costs about $300,000 a year per patient and although around 125 Australians are eligible for it, the cost to the government could be up to $38 million a year if it is PBS listed.

Professor Dominic Fitzgerald, a paediatric respiratory physician at the Children